The US Food and Drug Administrationdrugs (FDA) has approved selexipag to treat adults with pulmonary arterial hypertension (PAH), a chronic and progressive lung disease that causes high pressure in the arteries that connect the heart to the lungs.
The drug, marketed as Uptravi by Actelion Pharmaceuticals, is an oral prostacyclin receptor (IP) agonist. It is indicated to delay disease progression and reduce the risk of hospital admission for PAH. Selexipag, which has been granted orphan drug designation by the FDA, acts by causing vasodilation, reducing elevated arterial pressure.
“Uptravi offers an additional treatment option for patients with pulmonary arterial hypertension,” says Ellis Unger, director of the Office of Drug Evaluation, in the FDA’s Center for Drug Evaluation and Research.
Selexipag’s safety and efficacy were established in a long-term clinical trial of 1,156 participants with PAH, treated for a median of 1.4 years. Patients received selexipag or placebo twice daily and were also allowed to receive background PAH-specific therapy, including an endothelin receptor antagonist (ERA) and phosphodiesterase-5 (PDE-5) inhibitor. At the start of the study, 80% of patients were receiving either an ERA, a PDE-5 inhibitor or a combination of the two.
The trial showed that selexipag reduced hospital admissions related to PAH and reduced the risk of disease progression compared with placebo. Common side effects observed in those treated with selexipag included headache, diarrhoea, jaw pain, nausea, muscle pain, vomiting, pain in an extremity and flushing.
Uptravi will be made available to patients in the United States in early January 2016, says Actelion. The company is also seeking approval in Europe, where the drug is under review by the European Medicines Agency.